A Phase II, Open-Label Extension Study Evaluating the Long Term Safety, Tolerability & Efficacy of Orally-Administered CYT387 in Primary Myelofibrosis or Post-Polycythemia Vera or Post-Essential Thrombocythemia Myelofibrosis
The myeloproliferative neoplasms (MPN), most notably polycythemia vera (PV), essential
thrombocythemia (ET), and primary myelofibrosis (PMF) are a diverse but inter-related suite
of clonal disorders of pluripotent hematopoietic stem cells (Tefferi et al., 2008). The MPN
share a range of biological, pathological, and clinical features including the relative
overproduction of one or more cells of myeloid origin, growth factor independent colony
formation in vitro, marrow hypercellularity, extramedullary hematopoiesis, spleno- and
hepatomegaly, and thrombotic and/or hemorrhagic diatheses (Tefferi et al., 2005).
This is a multi centre, open-label, extension study of the core study (CCL09101). The
primary aims of the study will be to determine the long term safety and tolerability of
orally-administered CYT387 when administered as a capsule dose, on a 28-day treatment cycle.
Following completion of the core study (CCL09101), patients who have tolerated the drug and
derived clinical benefit will continue to be treated with CYT387 administered orally.
Subjects will be evaluated every three months for up to 24 cycles of CYT387 treatment.
Subjects will return for a follow-up visit 30 days after completion of the last dose of
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
To determine the long term safety and tolerability of orally-administered CYT387 in patients with PMF or post-ET/PV MF following completion of core study CCL09101
Safety monitoring will be undertaken for all patients every 3 months
Ayalew Tefferi, MD
United States: Food and Drug Administration
|Mayo Clinic||Rochester, Minnesota 55905|
|Dana-Farber Cancer Institute||Boston, Massachusetts 02115|
|Stanford Cancer Center||Stanford, California 94305-5824|